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ABSTRACT: Background
Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by muscle atrophy and weakness. SMA type 1 (SMA1) is the most severe form: affected infants are unable to sit unaided; SMA type 2 (SMA2) children can sit, but are not able to walk independently. The Standards of Care has improved quality of life and the increasing availability of disease-modifying treatments is progressively changing the natural history; so, the clinical assessment of nutritional status has become even more crucial. Aims of this multicenter study were to present the growth pattern of treatment-naïve SMA1 and SMA2, and to compare it with the general growth standards.Results
Body Weight (BW, kg) and Supine Length (SL, cm) were collected using a published standardized procedure.
SUBMITTER: De Amicis R
PROVIDER: S-EPMC8418717 | biostudies-literature | 2021 Sep
REPOSITORIES: biostudies-literature